Knowledge and perception of off-label drug use amongst prescribing physicians in a tertiary care hospital

1 Introduction

Off-label drug use is the administration of a drug, biologic or device for conditions outside the product license with respect to the dose, age, route of administration, indication or contra-indication [1, 2]. Off-label drug use is not illegal and has been reported to an extent of around 7.5 to 40% in adults [3] and may be up to 90% in children [4]. Off-label prescribing could be related to several problems such as safety for the patient, costs for the society, and legal risk for the physician; therefore, this practice should be used only when the benefit outweighs the risk [5]. Even studies have shown that there is a significant increase in the adverse events following off-label drug use [6, 7]. Studies coming from the developed nations have shown that around 30% of the prescribers are unaware of the concept of off-label drug use [8]. There have been no studies from India, a developing nation with varied practice of medicine systems from the western countries and so the present study was envisaged to explore the awareness and views of off-label drug use.

2 Methods

The study was initiated after obtaining permission from the institutional ethics committee and informed consent from all the study participants. A questionnaire to assess the above mentioned objective was created and validated by a group of experts in the qualitative research. The reliability of the questionnaire was assessed by repeated administering of the questionnaire over a span of two weeks (kappa = 0.87). Interns, junior residents and faculty members of various medical and surgical departments of Subharti Medical College, Meerut, India, a tertiary care teaching hospital were recruited in the present study. Descriptive analysis has been used for analyzing various categorical variables. As there has been no previous study done to evaluate this hypothesis, no formal sample size calculation was done.

3 Results

4 Discussion

The present study was conducted to evaluate the awareness of off-label drug use amongst theprescribing physicians in a tertiary care hospital in India and we found that 72% were aware of the concept of off-label drug use, though 48% felt that such use was illegal. Only 56% of the study participants inform the family members about prescribing off-label drugs while only 21% felt that the evidence was appropriate for using off-label drugs and 29% felt that their knowledge was adequate as far as the off-label drug use was concerned. A total of 69% of the study participants have used a drug in off-label manner and maximum (36.5%) did for indications.

Off-label drug use is not rare. Studies have reported such use ranging between 21 and 79% [9–14] in different populations (Table 2). The largest study on prevalence of off-label drug use was from Radley et al. [9] where 150 million prescriptions were assessed of which 21% were found to be off-label and most of them were related to cardiac medications (46%) followed by anticonvulsants (46%). Prescribing a drug in an off-label manner has several advantages in terms of enhancement of scientific knowledge especially when no other alternative exists or in case of “orphan” conditions [15]. At the same time, when the prescription is not based on evidence, it increases the risk of harms as was assessed by Radley et al. [9] where nearly two-thirds (73%) of the off-label prescriptions did not have any scientific basis. It is important for a prescriber to have adequate knowledge regarding use of drugs in an appropriate way viz. correct dose, indication, route and contra-indications. A study from 600 physicians in the United States (US) revealed that 45% were not aware of the US Food and Drugs Administration approval status of drugs for various indications [16]. Even in our study, we found that 77% of the study participants had inadequate knowledge. Focused group discussions and regular training of all the prescribers regarding off-label drug use is mandatory to indulge the practitioners in rational drug use.

A total of 39/70 (55.7%) study participants would inform the relatives while prescribing an off-label drug to the patients. An earlier study from the same population by Bang et al. [17] that had assessed the awareness of off-label drug use in parents found out that 93% of the parents wanted to be informed whenever doctor prescribes an off-label drug. Today in the era of treatment being taken primarily on the basis of shared decision-making, it is important to discuss the risks and benefits associated with such off-label drug use with the patient’s relatives as in the same study, 73% of the parents felt such use as illegal. Even in the present study, nearly half (41/85, 48.2%) of the doctors felt the same. Although no consensus has been achieved between various lawsuits regarding disclosure of off label use with the patient’s relatives, it is imperative that having had such discussion with the patient’s relatives shall reduce the chances of legal issues that may arise later on [18].

To conclude, we found an inadequate knowledge regarding the off-label drug use amongst the prescribers in a tertiary care medical college hospital. Many of the physicians felt such use as illegal and do not inform the patient’s relatives about such act. Considering the legal issues, clearly there exists need to patch up this lacuna in the developing countries like India.

Conflict of interest

The authors do not have any conflict of interest.